Biogen Acquires New Experimental SMA Therapy BIIB115 From Ionis
Biogen has exercised an option to acquire BIIB115, an investigational treatment for spinal muscular atrophy (SMA), from Ionis…
Articles by Marisa Wexler, MS
SMA Patients Value Medicines That Improve Motor Function, Study Says
When it comes to treatments, people with spinal muscular atrophy (SMA) types 2 and 3 place a lot of…
Spinraza Plus Zolgensma Offers Little Extra Benefit in Type 1, Study Suggests
Combined treatment with Spinraza and Zolgensma does not markedly improve motor function or breathing ability in children with spinal…
Treatment Delays May Be Less Important Than Family Support
The COVID-19 pandemic led to delays in treatment with Spinraza (nusinersen) for many children with spinal muscular atrophy (SMA).
Weak Trunk Muscles, Spine Problems Need Early Attention in SMA
Children with spinal muscular atrophy (SMA) who have spine deformities or weak trunk muscles are more likely than others…
Novartis Wraps Zolgensma Price, Availability Negotiations in Canada
Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its…
Newborn Screening Pilot for SMA in Belgium Grows Into Official Program
A pilot newborn screening (NBS) program for spinal muscular atrophy (SMA) that started in Liège province has transitioned into an…
Phase 3 Apitegromab Trial Planned for Non-ambulatory Types 2, 3
Scholar Rock plans to soon initiate a Phase 3 clinical trial of apitegromab, its investigational muscle-directed treatment for …
Pre-symptomatic Infants Retain Swallowing Ability in Evrysdi Trial
After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA)…
Variant in Androgen Receptor Might Be Useful in Treating SBMA
A naturally occurring form of the androgen receptor protein — which scientists have named AR45 because of its…